First Patient Dosed in Phase 2 Epidermolysis Bullosa Clinical Trial

According to a story from Market Screener, the drug developer RegeneRx Biopharmaceuticals, Inc. has recently announced that its licensee for RGN-137, an experimental wound healing gel, has begun the dosing of patients in a phase 2 clinical trial. This clinical trial is testing RGN-137 as a treatment for epidermolysis bullosa (EB), a rare disease that affects the skin. RegeneRx is focused on therapies for tissue protection, repair, and regeneration.

About Epidermolysis Bullosa (EB)

Epidermolysis bullosa is a group of genetic conditions which are most characterized by unusual blistering of the mucous membranes and skin. These painful blisters can occur with only minor friction or abrasion. The disease can be fatal in severe cases. These disorders are all caused by genetic mutations; the various subtypes of epidermolysis bullosa are classified by which genes are affected. Painful mucosal and skin blisters are the defining symptoms, but other complications may include esophageal narrowing and a vastly increased risk of skin cancer. There is no cure for the condition; treatment primarily focuses on symptom management, such as controlling pain, cleaning wounds, prevention or treatment of complications, nutritional support, and controlling infections. Males and females are affected at equal rates. The disease can seriously shorten lifespan, and in severe cases, patients do not survive beyond infancy. There is a dire need for more effective treatment approaches for epidermolysis bullosa. To learn more about these disorders, click here.

Changing Standards for Treatment

Recently, the US Food and Drug Administration (FDA) has issued specific ordinances regarding the development of treatments for epidermolysis bullosa. Previously, a drug could only provide complete wound healing to gain approval. Unfortunately, this standard has proved to be exceedingly difficult to reach. The new rules will allow drugs that can achieve partial wound healing to be considered for approval. This change will hopefully mean that more companies will begin working on therapies for this rare disease.

The clinical trial is unique in that all epidermolysis bullosa patients will receive the treatment. However, the study design still allows for controls to be maintained. Each patient will receive a placebo on one wound and RGN-137 on another. The wounds selected will be at a similar stage of healing (or lack thereof). 

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